On May 2, 2023 Kelonia Therapeutics, a biotech company revolutionizing in vivo gene delivery, reported that it will share initial preclinical data on its in vivo Gene Placement System (iGPS) technology during an oral presentation at the American Society of Gene & Cell Therapy (ASGCT) (Free ASGCT Whitepaper) 26th Annual Meeting, occurring May 16-20, 2023, in Los Angeles (Press release, Kelonia Therapeutics, MAY 2, 2023, View Source [SID1234630872]).

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The preclinical data presented will demonstrate that Kelonia’s iGPS technology uniquely enables safe and efficient in vivo generation of highly efficacious CAR T cells. In a humanized mouse model of multiple myeloma treatment, when an anti-BCMA CAR transgene was delivered directly in vivo using iGPS particles, low intravenous dose levels resulted in complete initial tumor regression comparable to ex vivo-manufactured CAR T cells. However, functional CAR T cell persistence after iGPS particle treatment resulted in significantly longer tumor control compared to ex vivo-generated CAR T cells. Despite efficient in vivo CAR gene transfer to T cells, no concerning or unexpected "off-target" transduction was observed including in vital, progenitor or reproductive organs.

"CAR T cells have been transformative for patients suffering from lethal cancers. But manufacturing unique CAR T cells for every patient has limited access to this incredible medicine. The preclinical data we’ll present at ASGCT (Free ASGCT Whitepaper) demonstrates the potential for potent, durable and safe CAR T cells generated by our iGPS in vivo delivery technology, without the requirement for preparative chemotherapy," said Kevin Friedman, Ph.D., Founder, President, and Chief Scientific Officer, Kelonia Therapeutics. "This is the first step toward our vision of solving the central challenge of genetic medicines: democratizing and enabling access when and where patients need them. We look forward to sharing detailed data at the conference."

Details for the oral presentation are as follows:

Abstract Title: Potent in vivo transduction by iGPS particles generates CAR T cells with durable anti-tumor activity

Presenter: Shannon Grande Contrastano, Ph.D., Senior Director, Preclinical Sciences, Kelonia Therapeutics

Session Date/Time: Wednesday, May 17, 2023: 3:45-5:30 PM PDT

Abstract number: 90

On May 2, 2023 Verismo Therapeutics, a clinical-stage CAR-T company and University of Pennsylvania spinout developing novel KIR-CAR platform technology, reported that it has activated its STAR-101 Phase 1 clinical trial at the University of Pennsylvania (Press release, Verismo Therapeutics, MAY 2, 2023, View Source [SID1234630870]).

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STAR-101 will evaluate Verismo’s lead candidate, SynKIR-110, an investigational new drug for the treatment of mesothelin-overexpressing malignant pleural mesothelioma, cholangiocarcinoma and ovarian cancer. Verismo Therapeutics is conducting a Phase 1 multicenter clinical trial in these tumor types to evaluate the feasibility and safety of SynKIR-110 (ClinicalTrials.gov Identifier: NCT05568680).

Verismo achieved clearance from the FDA to administer this multicenter clinical trial under the IND Verismo submitted for SynKIR-110, and Orphan Drug Designation for the treatment of mesothelin-expressing mesotheliomas with SynKIR-110 in September 2022. SynKIR -110 received Fast Track designation in April 2023.

"This trial marks a pivotal moment for our company," said Dr. Bryan Kim, Co-Founder and CEO of Verismo Therapeutics. We are grateful for the opportunity to partner with distinguished researchers at the University of Pennsylvania, including Dr. Janos Tanyi, MD, PhD, Dr. Andrew Haas, MD, PhD, and Dr. Mark O’Hara, MD, to bring this innovative therapy to those who require it the most."

Dr. Tanyi, an associate professor of Obstetrics and Gynecology in the Perelman School of Medicine at the University of Pennsylvania, and a principal investigator for the trial, said, "Our team is excited to be part of this clinical trial with Verismo. We are enthusiastic about the prospects of this innovative therapy and its capacity to provide new hope and improved outcomes for individuals who urgently need effective treatment options."

For more information about the STAR-101 clinical trial, please visit ClinicalTrials.gov NCT05568680.

About the KIR-CAR Platform
The KIR-CAR platform is a dual-chain CAR T cell therapy and has been shown in preclinical animal models to be capable of maintaining antitumor T cell activity even in challenging solid tumor environments. DAP12 acts as a novel costimulatory molecule for T cells using additional T cell stimulating pathways, further sustaining chimeric receptor expression and improving KIR-CAR T cell functional persistence. This continued T cell function and persistence can lead to ongoing regression of solid tumors in preclinical models, including those resistant to traditional CAR T cell therapies. The KIR-CAR platform is being investigated in combination with many additional emerging technologies, such as in vivo gene engineering, advanced cell manufacturing and reprogramming, combinational therapies, and even allogeneic cellular therapies to potentially provide the next-generation multimodal targeted immunotherapy for patients in need.

On May 2, 2023 EirGenix, Inc. (6589.TT) reported on 28th of April that it has completed the phase 1 clinical trial (EGC101) of its second breast cancer biosimilar EG1206A in Europe (Press release, EirGenix, MAY 2, 2023, View Source [SID1234630869]). Comparing EG1206A to Roche’s Perjeta US and EU, results of the clinical data analysis show that the main test indicators have met the pharmacokinetics bioequivalence standard. EG1206A is a biosimilar drug of the second-generation HER2-positive antibody drug Perjeta (pertuzumab). EG1206A has a unique binding mechanism for HER2 receptors which can produce a dual blockade effect. In combination with the first-generation HER2-positive antibody drug Trastuzumab, Pertuzumab has a synergistic effect in the treatment of early-stage HER2-positive breast cancer and metastatic breast cancer.

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The success of EG1206A’s phase 1 clinical trial is an important milestone for EirGenix and its development of biosimilar drug products for the treatment of HER2-positive breast cancer. Following the product launch of its first biosimilar drug EG12014 (trastuzumab biosimilar), EG1206A will soon begin its multi-country, multi-center global phase 3 clinical trials. After the initiation of the phase 3 clinical trials, it is expected that the evaluation of the primary end point indicators will be completed within two (2) years. Concurrently, EirGenix has been negotiating with global pharmaceutical companies for licensing marketing rights of EG1206A to further fast-track the launch of the biosimilar into the global market. Such achievements in EirGenix’s product development bring great excitement to the future of Taiwan’s biotechnology industry.

1. About EG1206A (pertuzumab biosimilar)

EG1206A is a biosimilar drug of the second-generation HER2-positive antibody drug Perjeta (pertuzumab). The clinical combination of trastuzumab and pertuzumab has a synergistic effect in the treatment of early-stage HER2-positive breast cancer and metastatic breast cancer. Patients with HER2-positive breast cancer account for approximately 20-30% of new breast cancer cases per year. The dual-drug therapy can help to prolong the survival period of breast cancer patients and is a standard treatment strategy recommended by many international treatment guidelines. According to 2022 sales data by Roche (developer of the originator drug Perjeta), global sales of Perjeta (pertuzumab) reached 4.087 billion Swiss Francs (4.56 billion U.S. Dollars). By the time the patent of Perjeta expires in 2026, it is expected that global sales will reach 6 billion U.S Dollars (approx. 180 billion New Taiwan Dollars). In 2021, Taiwan’s National Health Insurance paid 1.02 billion New Taiwan Dollars for Perjeta (pertuzumab) products. EirGenix’s EG1206A currently ranks amongst the top globally for most advanced developments of pertuzumab biosimilars. Once EirGenix’s products are launched onto the market, HER2-positive breast cancer patients will be provided with more accessible and affordable treatment options.

On May 2, 2023 Janssen Biotech, Inc., one of the Janssen Pharmaceutical Companies of Johnson & Johnson, reported that it has entered into a worldwide collaboration and license agreement with Cellular Biomedicine Group Inc. (CBMG) to develop, manufacture and commercialize next-generation chimeric antigen receptor (CAR) T-cell therapies for the treatment of B-cell malignancies (Press release, Janssen Biotech, MAY 2, 2023, View Source [SID1234630868]). These investigational CD20-directed autologous CAR-Ts have demonstrated promising overall and complete response rates in Phase 1 studies in patients with relapsed/refractory non-Hodgkin’s lymphoma (NHL) in China, with the majority of study participants having diffuse large B-cell lymphoma (DLBCL), the most common type of aggressive lymphoma accounting for approximately one-third of B-cell lymphomas globally.1,2

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DLBCL is characterized by the uncontrolled rapid growth of a type of immune cell called lymphocytes.3 CD20 and CD19 are antigens commonly found on the surface of the cells.4 As many as half of patients with DLBCL eventually become refractory to first-line treatment and require additional treatment options.5 C-CAR039 is a novel bispecific CAR-T therapy targeting both CD19 and CD20 antigens and has received U.S. Food and Drug Administration (FDA) Investigational New Drug (IND) clearance, and Regenerative Medicine Advanced Therapy and Fast Track designations for the treatment of patients with relapsed or refractory (R/R) DLBCL. A Phase 1b study in the U.S. evaluating C-CAR039 in the treatment of patients with R/R DLBCL is underway. C-CAR066 is an optimized novel CD20 targeted CAR-T therapy that has also received U.S. FDA IND clearance, and a Phase 1b study in patients with R/R DLBCL is anticipated to begin in the second half of 2023.

"We are committed to advancing the science and treatment of B-cell malignancies, especially in DLBCL where deeper responses and long-term remissions represent a persistent unmet need," said Sen Zhuang, M.D., Ph.D., Vice President, Clinical Research and Development, Janssen Research & Development, LLC. "A tenet to our continued innovation is a focus on accelerating the development of cell therapies as we strive to profoundly transform patient outcomes and, ultimately, progress potentially curative regimens."

Through the collaboration, Janssen enhances its portfolio in B-cell malignancies and strengthens its more than two-decade legacy in hematology, while deepening its commitment to accelerate development, manufacturing and commercialization capabilities to deliver best-in-class cell therapies. Under terms of the agreement, CBMG will grant Janssen a worldwide license to develop and commercialize the CAR-T assets, except in Greater China. Janssen and CBMG will negotiate an option for Janssen to commercialize the products in the China territory. Janssen will make an upfront payment of $245 million that will be accounted for in the second quarter as a research and development expense. Additional future payments will be based upon the achievement of certain development, regulatory and sales milestones, as well as tiered royalty payments on worldwide net trade sales, excluding Greater China.

Johnson & Johnson estimates that this collaboration, licensing agreement and development program will have an annual approximate 10 cent negative impact on earnings per share (EPS) in 2023 and 2024. The charge will largely apply to the second quarter and the company is maintaining its 2023 guidance.

The closing of the transaction is subject to customary closing conditions, including clearance under the Hart-Scott-Rodino Antitrust Improvements Act, and is expected to close in the second quarter of 2023.

"Our innovation strategy is agnostic to the source of breakthrough science, platforms, targets and medicines from the global life science ecosystem," said Yusri Elsayed, M.D., M.HSc., Ph.D., Vice President, Disease Area Leader, Hematologic Malignancies, Janssen Research & Development, LLC. "The Cellular Biomedicine Group team has discovered differentiated cell therapies with clinically validated CD20 CAR constructs, and we look forward to harnessing our expertise, capabilities and scale to lead the global development of these innovative CAR-T products."

About B-cell Malignances
B-cell malignancies, or B-cell lymphomas, are a type of cancer that arise from B cells (a type of immune system cell). Most B-cell lymphomas are NHL, and DLBCL is the most common. Globally, it is estimated that more than 540,000 new cases of NHL occurred in 2020, and more than 259,000 people died from the disease.6,7 DLBCL is most common in the U.S. and Western Europe, and the incidence has increased by more than 50 percent in 20 countries since the late 20th century.2 Other types of B-cell malignancies include Burkitt lymphoma, mantle cell lymphoma (MCL), and chronic lymphocytic leukemia/small lymphocytic lymphoma (CLL/SLL). Prognosis and treatment depend on type and stage of the disease.

On May 2, 2023 Jazz Pharmaceuticals plc (Nasdaq: JAZZ) reported that the Company will participate in the 2023 RBC Capital Markets Global Healthcare Conference (Press release, Jazz Pharmaceuticals, MAY 2, 2023, View Source [SID1234630867]). Company management will participate in a fireside chat on Tuesday, May 16, 2023, at 4:05 p.m. ET / 9:05 p.m. IST.

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An audio webcast of the fireside chat will be available via the Investors section of the Jazz Pharmaceuticals website at www.jazzpharmaceuticals.com. A replay of the webcast will be archived on the website for 30 days.